JP5829372B2 - 神経代謝性疾患のための遺伝子治療 - Google Patents

神経代謝性疾患のための遺伝子治療 Download PDF

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JP5829372B2
JP5829372B2 JP2008510224A JP2008510224A JP5829372B2 JP 5829372 B2 JP5829372 B2 JP 5829372B2 JP 2008510224 A JP2008510224 A JP 2008510224A JP 2008510224 A JP2008510224 A JP 2008510224A JP 5829372 B2 JP5829372 B2 JP 5829372B2
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mice
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JP2008540445A5 (enExample
JP2008540445A (ja
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マルコ・エイ・パッシーニ
ジェイムズ・ダッジ
グレゴリー・アール・スチュアート
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    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

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JP2008510224A 2005-05-02 2006-05-02 神経代謝性疾患のための遺伝子治療 Active JP5829372B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US67705705P 2005-05-02 2005-05-02
US60/677,057 2005-05-02
US68580805P 2005-05-31 2005-05-31
US60/685,808 2005-05-31
PCT/US2006/017242 WO2006119458A1 (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

Related Child Applications (1)

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JP2013201138A Division JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療

Publications (3)

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JP2008540445A JP2008540445A (ja) 2008-11-20
JP2008540445A5 JP2008540445A5 (enExample) 2009-07-09
JP5829372B2 true JP5829372B2 (ja) 2015-12-09

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JP2008510224A Active JP5829372B2 (ja) 2005-05-02 2006-05-02 神経代謝性疾患のための遺伝子治療
JP2013201138A Pending JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療
JP2015197051A Active JP6338560B2 (ja) 2005-05-02 2015-10-02 神経代謝性疾患のための遺伝子治療

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JP2013201138A Pending JP2014037418A (ja) 2005-05-02 2013-09-27 神経代謝性疾患のための遺伝子治療
JP2015197051A Active JP6338560B2 (ja) 2005-05-02 2015-10-02 神経代謝性疾患のための遺伝子治療

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US (2) US10632213B2 (enExample)
EP (4) EP3058959B1 (enExample)
JP (3) JP5829372B2 (enExample)
CN (4) CN107007842A (enExample)
AT (1) ATE525092T1 (enExample)
AU (1) AU2006243776A1 (enExample)
BR (1) BRPI0611379A2 (enExample)
CA (2) CA2998603C (enExample)
ES (1) ES2887076T3 (enExample)
IL (2) IL187078A (enExample)
PL (4) PL2420256T3 (enExample)
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WO (1) WO2006119458A1 (enExample)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3302529B2 (ja) 1995-04-28 2002-07-15 株式会社クボタ ポンプ吸水槽の整流装置
MX2007013734A (es) * 2005-05-02 2008-03-14 Genzyme Corp Terapia genica para trastornos de la medula espinal.
CN107007842A (zh) 2005-05-02 2017-08-04 建新公司 神经代谢疾病的基因治疗
ES2596885T3 (es) 2006-06-07 2017-01-12 Genzyme Corporation Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la medula espinal
PT3252161T (pt) * 2007-06-06 2022-02-01 Genzyme Corp Terapia genética para doenças do armazenamento lisossomal
EP2352847B1 (en) 2008-11-10 2014-01-08 The United States of America, as represented by The Secretary, Department of Health and Human Services Gene signature for predicting prognosis of patients with solid tumors
RS67077B1 (sr) 2009-05-02 2025-08-29 Genzyme Corp Genska terapija za neurodegenerativne poremećaje
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
WO2012145646A1 (en) * 2011-04-20 2012-10-26 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016081924A1 (en) * 2014-11-20 2016-05-26 Duke University Compositions, systems and methods for cell therapy
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
AU2016219398A1 (en) 2015-02-10 2017-09-28 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex
US11046746B2 (en) 2015-03-26 2021-06-29 Suzhou Auzone Biological Technology Co., Ltd Method of treatment of cerebral amyloid angiopathy with P75ECD peptide and/or P75ECD-FC fusion protein
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2016196507A1 (en) * 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
CN110809583A (zh) 2017-06-07 2020-02-18 瑞泽恩制药公司 用于内化酶的组合物和方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
KR20200118151A (ko) 2018-02-07 2020-10-14 리제너론 파마슈티칼스 인코포레이티드 치료 단백질 전달을 위한 방법 및 조성물
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
MX2020012350A (es) 2018-05-17 2021-01-29 Regeneron Pharma Anticuerpos anti-cd63, conjugados y usos de estos.
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
KR20240126916A (ko) * 2023-02-14 2024-08-22 경북대학교 산학협력단 Asm 단백질 또는 이의 단편을 포함하는 퇴행성 신경질환 또는 우울증 예방 또는 치료용 조성물

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE8303626D0 (sv) 1983-06-23 1983-06-23 Kabigen Ab A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
JP3532566B2 (ja) 1993-06-24 2004-05-31 エル． グラハム，フランク 遺伝子治療のためのアデノウイルスベクター
JP3875990B2 (ja) 1993-10-25 2007-01-31 カンジ，インコーポレイテッド 組換えアデノウイルスベクターおよび使用方法
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
WO1997039789A1 (en) 1996-04-22 1997-10-30 Medtronic, Inc. Two-stage angled venous cannula
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
US6582692B1 (en) * 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US20030165481A1 (en) * 2000-02-24 2003-09-04 Hersh Louis B. Amyloid peptide inactivating enzyme to treat Alzheimer's disease
EP1319082B1 (en) 2000-09-18 2005-11-16 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
US20030050273A1 (en) * 2001-08-29 2003-03-13 Keiya Ozawa Compositions and methods for treating neurodegenerative diseases
US7232670B2 (en) * 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
NZ533833A (en) 2001-12-21 2008-01-31 Salk Inst For Biological Studi Targeted retrograde gene delivery to motor neurons
US6998118B2 (en) 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
AU2003220115A1 (en) * 2002-05-20 2003-12-12 Board Of Regents, The University Of Texas System Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
PL1620133T3 (pl) * 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
US7740168B2 (en) 2003-08-18 2010-06-22 Visa U.S.A. Inc. Method and system for generating a dynamic verification value
AU2003272868A1 (en) 2003-10-15 2005-04-27 Agtc Gene Technology Company Ltd. Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors
EP1716870A1 (en) 2005-04-29 2006-11-02 Bracco Imaging S.p.A. MRI contrast agents endowed with concentration independent responsiveness
CN107007842A (zh) 2005-05-02 2017-08-04 建新公司 神经代谢疾病的基因治疗
MX2007013734A (es) 2005-05-02 2008-03-14 Genzyme Corp Terapia genica para trastornos de la medula espinal.
AR059371A1 (es) 2006-02-08 2008-03-26 Genzyme Corp Terapia genica para la enfermedad de niemann-pick tipo a
EP2709991B1 (en) 2011-05-16 2020-09-02 Genzyme Corporation Use of cxcr4 antagonists for treating WHIM syndrome, myelokathexis, neutropenia and lymphocytopenia
TWI821227B (zh) 2017-12-22 2023-11-11 美商健臻公司 胺甲喋呤誘發的免疫耐受性之生物標記

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EP1879624A1 (en) 2008-01-23
EP1879624A4 (en) 2009-03-25
PT3520823T (pt) 2021-09-16
JP2014037418A (ja) 2014-02-27
JP2008540445A (ja) 2008-11-20
EP3520823B1 (en) 2021-06-30
WO2006119458A1 (en) 2006-11-09
CA2607173A1 (en) 2006-11-09
PL1879624T3 (pl) 2012-02-29
IL239167A0 (en) 2015-07-30
PL2420256T3 (pl) 2016-12-30
CN101212988A (zh) 2008-07-02
CA2607173C (en) 2018-04-24
CA2998603C (en) 2019-07-23
ATE525092T1 (de) 2011-10-15
CN107007842A (zh) 2017-08-04
PT2420256T (pt) 2016-09-13
EP3058959A1 (en) 2016-08-24
IL187078A (en) 2015-06-30
CN107362371A (zh) 2017-11-21
JP6338560B2 (ja) 2018-06-06
PT3058959T (pt) 2019-05-24
US20080292593A1 (en) 2008-11-27
WO2006119458A9 (en) 2007-02-15
US10632213B2 (en) 2020-04-28
ES2887076T3 (es) 2021-12-21
EP2420256B1 (en) 2016-06-22
US11957765B2 (en) 2024-04-16
AU2006243776A1 (en) 2006-11-09
IL187078A0 (en) 2008-02-09
US20200306387A1 (en) 2020-10-01
EP1879624B1 (en) 2011-09-21
EP3520823A1 (en) 2019-08-07
EP3058959B1 (en) 2019-01-30
PT1879624E (pt) 2012-02-02
BRPI0611379A2 (pt) 2010-08-31
JP2016041706A (ja) 2016-03-31
US20140356327A9 (en) 2014-12-04
CA2998603A1 (en) 2006-11-09
PL3058959T3 (pl) 2019-07-31
EP2420256A1 (en) 2012-02-22
CN104306986A (zh) 2015-01-28
PL3520823T3 (pl) 2021-11-22

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